Muscle Study Group Annual Meeting Program
2017
Muscle Study Group Annual Meeting Program
2017
02 Welcome from Richard Barohn, M.D. and Prof Michael Hanna 03 List of MSG Committees 04 Meeting Amenities and Registration Information 05 Official Networking Events 06 CME Accreditation Information 07 Speaker and Poster Information 08 Agenda 11
Meeting Support
12
Cliff Lodge Floorplan Level B
13
Cliff Lodge Floorplan Level C
14 Village Map
01 Table of Contents
01 Table of Contents
02 Welcome from Richard Barohn, M.D. and Prof Michael Hanna
Dear Colleagues: On behalf of your Muscle Study Group co-chairs we would like to welcome each of you to the 2017 Muscle Study Group Annual Meeting in Snowbird, Utah. It is an exciting time in neuromuscular research as we continue to grow and adapt. The world of neuromuscular research is a fascinating area in which to work and study, and we’ll continue to meet and bring inspired people together in meetings like this to ensure the Muscle Study Group remains at the cutting edge of sophisticated approaches to therapy. We would like to give you an idea of what you can expect and what we hope to achieve over the next few days. The conference will cover neuromuscular therapeutics: bench to bedside and beyond. This year’s conference has five main sessions – Session I: Platforms for Developments of Novel Therapeutics; Session II: Data Mining in Neuromuscular Therapeutics; Session III: Outcome Measures in Neuromuscular Disease; Session IV: Post-Marketing Surveillance in Neuromuscular Disease; and Session V: Identification of Neuromuscular Disease. Throughout these three days there will be rich programming, targeted training and numerous networking opportunities. The workshop session will focus on early career development and how to get started. We are excited to have Brian K. Kaspar, Ph.D., from Nationwide Children’s Hospital here with us to give the Robert C. Griggs Annual MSG keynote speech. As the co-chairs of the Muscle Study Group, we would like to thank this year’s planning committee for putting together an excellent agenda that covers such a broad range of topics and interests within the neuromuscular field. We would also like to thank Snowbird for allowing us to hold our annual meeting at their resort for the second year in a row. The staff here at Snowbird plays a huge role in making our annual meetings so successful. We hope that you all enjoy all wonderful amenities that the resort has to offer. Before we close, I’d like to thank each of your for attending our conference and bringing your expertise to our gathering. You have vision, the knowledge and the experience to help us pave our way into the future. You are truly the greatest asset today and tomorrow, and we could not accomplish what we do without your support and leadership. Throughout this conference, we ask you to stay engaged, keep us proactive and help us shape the future of neuromuscular research. Our thanks goes out to all of you. Best wishes,
Richard J. Barohn, M.D. Chairman and Professor of Neurology, KUMC Co-chairman of the Muscle Study Group
Prof Michael G. Hanna Director, UCL Institute of Neurology Co-chairman of the Muscle Study Group
• Richard Barohn, M.D., Chair University of Kansas Medical Center • Michael Hanna, M.D., Co-Chair University College London • Robert Griggs, M.D., Former Chair University of Rochester Medical Center • Tahseen Mozaffar, M.D., Investigator Member UC Irvine • Mazen Dimachkie, M.D., Investigator Member University of Kansas Medical Center • Emma Ciafaloni, M.D., Investigator Member University of Rochester Medical Center • Heidi Runk, Coordinator Member Penn State University • Lindsay Alfano, Evaluator Member Nationwide Children’s Hospital • Michael McDermott, Ph.D., Biostatistician University of Rochester Medical Center • Rabi Tawil, M.D., Director MSG Coordination Center University of Rochester Medical Center
2017 MSG Annual Meeting Planning Committee • • • • • •
Nicholas Johnson, M.D. University of Utah Health Care Jeffrey Statland, M.D. University of Kansas Medical Center Pedro Machado, M.D. University College London Jaya Trivedi, M.D. UT Southwestern Medical Center Chris Weihl, M.D. Washington University Physicians Chad Heatwole, M.D. University of Rochester Medical Center
Nominating Committee • John Kissel, M.D., Chair Ohio State University Wexner Medical Center • Nicholas Johnson, M.D. University of Utah Health Care • Laura Herbelin, University of Kansas Medical Center
Constitution Committee • Jaya Trivedi, M.D., Chair UT Southwestern • Shannon Venance, M.D. London Health Science Centre • Alejandro Tobon-Gonzalez, M.D. UT Health Science Center in San Antonio
Fellowship Committee • Gordon Smith, M.D., Chair University of Utah Health
03 List of MSG Committees
MSG Executive Committee
Check-In and Registration Information
04 Meeting Amenities and Registration Information
Located in the lobby of the main meeting hall Saturday, September 23, 2017 7:30-8:00 a.m. 1:00-1:30 p.m. 7:00-7:30 p.m.
Sunday, September 24, 2017 7:30-8:00 a.m. 10:30-11:00 a.m. 6:45-7:00 p.m.
Note: Early arrival check-in will be available on Friday, September 22, 2017 from 5:30-6:30 p.m. on Level C at the top of the escalators.
Registration Materials • Name badge • Program book • MSG/USB drive/bracelet –– PDF of program booklet –– Speaker abstracts and bios (those we were given permission to provide) –– PDF Muscle & Nerve journal –– Attendee list • 2017 MSG edition of the Muscle & Nerve journal
Attendee Help Desk/Snowbird Tourism Information Please visit concierge located on Level C for all information regarding Snowbird. Please note that concierge will also help you set up your return shuttle to the airport following the meeting.
Dining The MSG is providing all meals. However, Snowbird offers many shops and restaurants as alternative options. Please see the village map on page 18-19 for more information.
Meeting Staff Staff members and volunteers are happy to assist with any questions attendees may have regarding the meeting or the Snowbird Center. Attendees can easily locate them by their bright yellow name badge lanyards.
Wireless Internet The MSG is providing free Wi-Fi:
Username: UKMC2017
Password: snowbird
Official Networking Events
Enjoy s’mores by the fireside on the Atrium Patio while networking with old and new friends and taking in all of Utah’s fresh mountain air. Our fireside events are always a hit so this isn’t something you will want to miss!
Sunday, September 24, 2017 Walk-through poster session and evening reception 7:00-10:00 p.m. Attendees are invited to the Primrose Room for a cocktail reception and the opportunity to mix and mingle amongst the poster submissions with young and experienced investigators, industry colleagues and friends while enjoying hors d’oeuvres and signature MSG beverages. The outdoor patio will be open for those who wish to enjoy the evening by the fire.
05 Official Networking Events
Saturday, September 23, 2017 Campfire and s’mores outdoor reception 8:00 p.m. Sponsored by CSL Behring
CME Accreditation Information
06 CME Accreditation Information
MSG 2017 will be CME accredited. Accreditation will be provided by the University of Kansas Medical Center Continuing Education and Professional Development. All participants are required to sign the attendance roster at check-in of the meeting. A certificate of completion will be provided to all activity participants based on completion of the program evaluation, documentation of actual attendance time, meeting minimum attendance requirements specific to the activity and payment in full. Following the meeting you will receive a survey via the email you provided with registration. Once the survey is complete you will receive your certificate of completion. Physicians: The University of Kansas Medical Center Office of Continuing Medical Education is accredited by the Accreditation Council for Continuing Medical Education to provide continuing medical education for physicians. The KU Medical Center Office of Continuing Medical Education designates this live activity for a maximum of 14.25 AMA PRA Category 1 Credits.TM Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Speakers/presenters: Please bring your presentation to the podium 15 minutes before the start of your session so that your slides can be loaded. Our technical staff will assist you with any audio/visual needs you may have. If needed, our staff will assist you with uploading your presentation. Please do not bring your own laptop as we will provide one for you.
Posters The poster exhibition is located in the Primrose Room. Walk Through Poster Session: Day 2, Saturday, September 24, 2017 from 7:00-10:00 p.m. Please set up your poster in its designated spot during breakfast on Day 2 from 7:00-8:00 a.m. Please see Kara Thompson at registration for further details. All posters will be displayed throughout the entire meeting. Important note: Poster presenters are requested to be beside their poster during the Walk Through Poster Session on Day 2.
07 Speaker and Poster Information
Speaker and Poster Information
Friday, September 22, 2017 We will have early check in from 6:00-7:00 p.m. located at the top of the escalators for those who arrive early on Friday.
Day 1: Saturday, September 23, 2017
8:00 p.m.
Day 2: Sunday, September 24, 2017 7:00-8:00 a.m.
08 Agenda
6:30-7:40 a.m.
Buffet breakfast and check-in Golden Cliff Room
7:45 a.m.5:00 p.m.
Study updates/workshops Ballroom
7:45-8:30 a.m.
Memantine update Richard J. Barohn, M.D.
8:30-9:00 a.m.
Rasagiline update Richard J. Barohn, M.D.
9:00-10:00 a.m.
IPNEMA update Tahseen Mozaffar, M.D.
10:00-10:40 a.m.
Arimoclomol update Mazen Dimachkie, M.D.
10:45-11:00 a.m.
IBM Gene Consortium Update Mike Hanna, M.D.
11:00 a.m.12:00 p.m. 12:00-1:30 p.m.
Campfire and s'mores outdoor reception Atrium Patio Sponsored by CSL Behring
Buffet breakfast Golden Cliff Room 2016 MSG planning committee meeting Magpie A Room At this time we ask for people to set up their posters in the Primrose Room for display.
8:00-8:30 a.m.
Welcome and state of the Muscle Study Group address Ballroom
8:30-10:10 a.m.
RRNMF John Katz, M.D.
Session I: DMD as a model of neuromuscular disease across the lifespan* Ballroom Moderator: Valeria Sansone, M.D., Ph.D.
8:35-9:05 a.m.
Lunch Golden Cliff Room
Early outcomes in DMD* Anne Connolly, M.D.
9:10-9:35 a.m.
Muscle Study Group executive committee meeting Magpie Room
Trial design in DMD: Lessons learned from natural history* Valeria Sansone, M.D., Ph.D.
9:40-10:05 a.m.
Modifiers in DMD* Robert B. Weiss, Ph.D.
1:00-2:00 p.m.
MG-SCIg update Mazen Dimachkie, M.D.
10:10-10:30 a.m.
Refreshments break Ballroom Lobby
2:00-3:00 p.m.
N103 Beat MG Richard Nowak, M.D.
10:30 a.m.12:45 p.m.
3:00-5:00 p.m.
Young investigator and underrepresented NM population workshop Ballroom
Session II: Extracellular Matrix Dysfunction as a disease mechanism* Ballroom Moderator: Russ Butterfield, M.D.
3:00-4:00 p.m.
Panel discussion Led by Nicholas Johnson, M.D. and Chris Weihl, M.D.
10:40-11:05 a.m.
4:00-5:00 p.m.
Break-out discussion session
The Myomatrix reloaded: Integrin and ECM therapies for the muscular dystrophies* Dean Burkin, Ph.D.
5:00-6:15 p.m.
Dinner Golden Cliff Room
11:10-11:35 a.m.
Dystrogylcanopathy Cohort* Katherine Mathews, M.D.
6:45 p.m.
Keynote speaker Ballroom Introduction by Chris Weihl, M.D.
11:40 a.m.12:05 p.m.
Collagen VI-related dystrophies: A translational update* Carsten Bonnemann, M.D.
Experiences in academic translational medicine* Steve Greenberg, M.D. *CME Credit
Lunch Golden Cliff Room
5:55-6:05 p.m.
Evaluating the benefits of community-based aerobic training on the physical health and well-being of people with neuromuscular disease Gita Ramdharry, Ph.D.
6:10-6:20 p.m.
RNA-sequenced gene expression profiling identifies up-regulation of novel muscle-specific gene musclin in adult and congenital myotonic dystrophy Don McCorquodale, M.D., Ph.D.
6:30-7:30 p.m.
Dinner Golden Cliff Room
7:40 p.m.
Robert C. Griggs Annual MSG keynote speaker Ballroom Introduction by Mike Hanna, M.D.
PCORI investigator meeting at this time Magpie B Room 1:30-3:05 p.m.
Session III: Non-coding repeats as a disease mechanism* Ballroom Moderator: Chris Weihl, M.D.
1:40-2:05 p.m.
Myotonic dystrophy as a paradigm for RNA gain function disease mechanisms* Thomas A. Cooper, M.D.
2:10-2:35 p.m.
Update on myotonic dystrophy trial readiness* Charles A. Thornton, M.D.
2:40-3:05 p.m.
Disease progression and pathogenesis in congenital myotonic dystrophy* Nicholas Johnson, M.D.
3:10-4:20 p.m.
Walk-through poster session Primrose Room
4:20-6:10 p.m.
Poster session presentations Ballroom Moderator: Valeria Sansone, M.D., Ph.D.
4:25-4:35 p.m.
Open label trial of Ranolazine in Myotonia Congenita W. David Arnold, M.D.
4:40-4:50 p.m.
PRISM-SMA: Patient reported impact of symptoms in adult Spinal Muscular Atrophy Chad Heatwole, M.D.
4:55-5:05 p.m.
5:10-5:20 p.m.
The clinical phenotype and neuropathic outcomes of Paclitaxel-Acute Pain Syndrome Summer Karafiath, M.D. Baseline characteristics of a prospective natural history study of sporadic inclusion body myositis including MRI assessment Pedro Machado, M.D.
5:25-5:35 p.m.
Limits of postural stability in Myotonic Dystrophy compared to normative values Mary Pautler, MPH
5:40-5:50 p.m.
Axonal RNA profiling of human motor neurons from patients with Charcot-MarieTooth disease as a novel approach to study axon degeneration Mario Saporta, M.D., Ph.D.
*CME Credit
Clinical trials in DMD 2016: Where are we?* Ballroom Francesco Muntoni, M.D. 9:00 p.m.
MSG Mentors and Young Investigator Reception Primrose Room
Day 3: Monday, September 25, 2017 7:00-8:00 a.m.
Breakfast Golden Cliff Room CReATe investigator meeting at this time Magpie A Room
8:10-9:55 a.m.
Session IV: Mechanisms of Chaperone dysfunction* Ballroom Moderator: Nicholas Johnson, M.D.
8:10-8:35 a.m.
Chaperone function and dysfunction in neuromuscular disease* Chris Weihl, M.D.
8:40-9:05 a.m.
Proteomic identification of aggregates and chaperones in disease muscle* Rudolf Kley, M.D.
09 Agenda
12:10-1:25 p.m.
10 Agenda
9:10-9:55 a.m.
Phase 2 trial results of autographic manipulation with trehalose in Oculopharyngeal Muscular Dystrophy* Zohar Argov, M.D.
10:00-10:30 a.m.
Refreshments break Ballroom Lobby
10:30 a.m.1:05 p.m.
Session V: Autoimmunity* Moderator: Jeffrey Guptill, M.D.
10:40-11:05 a.m.
Mechanisms of Autoimmunity* Mark Anderson, M.D.
11:10-11:35 a.m.
MGTX: Results from the thymectomy trial in non-thymomatous myasthenia gravis* Gil Wolfe, M.D.
11:40 a.m.12:05 p.m.
Outcome measures in autoimmune NMJ diseases* Renato Mantegazza, M.D.
12:10-12:35 p.m.
2:40- 3:20 p.m.
Hot topics session Ballroom Zika virus update saliva Osvaldo Nascimento, M.D. REGAIN: A phase-2 randomized, double-blind, placebo-controlled, multicenter study to evaluate the safety and efficacy if Eculizumab in subjects with refractory generalized Myasthenia Gravis James Howard, M.D.
3:20-4:40 p.m.
Session VI: Mentoring for young investigators and underrepresented NM population to promote increased engagement in neuromuscular research* Ballroom Moderator: Gordon Smith, M.D.
Targeting FcRN in neuromuscular disease* Jan L. Hillson, M.D.
3:30-3:50 p.m.
NIH programs for young investigators* Glen Nuckolls, Ph.D.
12:40-1:05 p.m.
AChR-specific immunosuppressive therapy for MG* Jon Lindstrom, Ph.D.
3:55-4:15 p.m.
Advice for young investigators* Richard J. Barohn, M.D.
4:20-4:40 p.m.
1:10-2:30 p.m.
Lunch Golden Cliff Room
Research support resources for junior investigators* Jeffrey Guptill, M.D.
4:50-6:50 p.m.
Break
7:00-8:15 p.m.
Dinner Golden Cliff Room
*CME Credit
Meeting Support The MSG would like to thank the following companies for their generous support:
Platinum Level
Gold Level NIH Grifols
Silver Level Alexion Alnylam AxelaCare Cytokinetics Idera Pharmaceuticals Option Care NuFactor
Bronze level Catalyst Amicus
11 Meeting Support
CSL Behring
10/12
Level B
The Cliff Lodge
Snowbird
AV 4
AV 1
AV 2
AV 3 AV 5
17 Venue Maps 12 Cliff Lodge Floorplan Level B
Level C
The Cliff Lodge
Snowbird
13 Cliff Lodge Floorplan Level C
ATM
Primrose B
Primrose A
Amphitheater Lobby Patio Event Tent Plaza Deck Skier’s Bridge
The Forklift Baked & Brewed General Gritts Tram Car Pizza Steak Pit The Tram Club
Snowbird Center
Wildflower Restaurant
Iron Blosam Lodge
The Lodge Bistro Restaurant & Lounge
Peruvian Chairlift & Tunnel Ropes Course Aerial Tram Alpine Slide Mountain Flyer Mountain Coaster Cool Air Concert Series Family Flicks Oktoberfest
Summer Activities
Fitness Room & Classes Roof Top Pool & Hot Tub Salon
The Atrium The Aerie El Chanate Restaurant & Cantina Superior Snacks
The Cliff Lodge
The Cliff Spa
The Cliff Lodge
Dining & Nightlife
Outdoor Meeting Venues The Lodge at Snowbird Conference Center Terrace Tent
The Cliff Lodge Meeting & Banquet Rooms Snowbird Center The Lodge at Snowbird
Meetings & Banquets
Iron Blosam Lodge
Village Map
Snowbird
14 Village Map
Mountain Flyer
Wine & Liquor Store Pipeline Center Sundries Christy Sports Superior Photography Snowbird Sports
Snowbird Center
Cliff Sundries Marco Polo’s The Cliff Spa Shop
The Cliff Lodge
Activity Center
Shops
Alpine Slide
Conference Center Terrace Tent
ATM Post Office Pharmacy Public Safety Adaptive Sports
Snowbird Center
Fire Station
ATM Public Safety
The Cliff Lodge
Services
Mountain Coaster
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